Key clinical point: A single infusion of SPK-8011 reduced bleed and infusion rates in severe hemophilia A patients.
Major finding: The overall reduction in annualized infusion rate (AIR) and annualized bleeding rate (ABR) was 97% for each.
Study details: A phase 1/2 study of 12 patients
Disclosures: Spark Therapeutics sponsored the study. Dr. George reported equity ownership related to the University of Pennsylvania and consultancy for Pfizer.
High K et al. ASH 2018, Abstract 487.
This Week's Must Reads
First-line avelumab/axitinib for RCC benefits wide range of patients, Choueiri TK et al. GUCS 2019, Abstract 544.
QC measures improve with CMS Oncology Care Model, Rocque G et al. J Oncol Pract. 2019. doi: 10. 1200/JOP.18.00390.
Women less likely to receive industry funds, Weng JK et al. JAMA Netw Open. 2019 Jan. 25. doi: 10.1001/jamanetworkopen.2018.7377.
Must Reads in Bleeding Disorders
Caplacizumab approved for adults with aTTP , FDA news release: FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder
IVIG versus anti-D in pediatric ITP , Lioger B et al. J Pediatr. 2019; 204:225-33.
Promising treatment for heavy menstrual bleeding , O’Brien SH et al. J Pediatr Adol Gynec. 2019 Feb 4. doi: 10.1016/j.jpag.2019.01.009.
Beware of ICH in severe hemophilia cases , Zwagemaker AF et al. EAHAD 2019, Abstract OR08.
75 exposure days is the magic number in inhibitor risk , van den Berg HM et al. EAHAD 2019, Abstract OR05.